Oligonucleotide-Based Therapeutics Conference

There is tremendous interest in developing effective and safe RNA-based therapeutics for the treatment of inherited muscle diseases. At least four RNA oligonucleotide drugs have received FDA accelerated approval; however, clinical benefit has yet to be determined. This session will navigate current and future landscapes of oligonucleotide-based therapeutic developments. The focus will be on defining hope, identifying challenges, and approaches to turn challenges into opportunities in treating disabling rare genetic muscle diseases. These diseases have presented with unprecedented pathogenesis and associated challenges for drug development. For example, exon skipping in Duchenne muscular dystrophy, nuclear RNA-protein aggregation in myotonic dystrophy, and inefficient repeat-mediated epigenetic repression of the DUX4 retrogene embedded in the D4Z4 repeat units in facioscapulohumeral dystrophy. Further, muscle as a target organ is tough to get the therapeutic RNA to go to when administered in bloodstream or subcutaneously. There are also challenges in the assessment of treatment response in these rare diseases necessitating development of biomarkers and endpoints, innovative clinical trial designs, and application in real world clinical practice.